Zilucoplan Treatment of Severe MG Exacerbations Leading to Hospitalization of Participants With Acetylcholine Receptor Antibody Positive gMG

What this Trial Is About

Researchers are evaluating the use of subcutaneous zilucoplan in people with acetylcholine receptor antibody positive generalized myasthenia gravis (gMG) who experience severe exacerbations that require hospitalization. The study aims to find a less invasive treatment option for severe symptoms like respiratory or bulbar dysfunction. This is an open-label, phase 3b interventional study involving about 15 patients to assess if zilucoplan can quickly improve these severe symptoms and reduce hospital stays. Participants will receive daily subcutaneous injections of zilucoplan for 12 weeks, with doses based on body weight. Treatment starts while patients are hospitalized and continues after discharge as outpatients. Patients have the option to undergo standard plasma exchange or intravenous immunoglobulin (IVIG) treatment, but this study focuses on evaluating zilucoplan as an alternative. Those treated with zilucoplan will receive meningococcal vaccinations before starting and continue with recommended vaccines for 6 months while taking antibiotics until fully vaccinated. During the approximately 18-week study period, participants will undergo assessments including medical history, physical exams, vital signs, blood tests, and specific myasthenia gravis evaluations like the Quantitative Myasthenia Gravis (QMG) score, MG-Activities of Daily Living (MG-ADL) score, and quality of life measures. Researchers will monitor current medications and adverse events. The main outcome measured is the change in MG-ADL score from baseline to week 2, with further assessments at week 12. Safety and treatment effects will be closely observed throughout the study.

Zilucoplan for Severe gMG Exacerbations